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Post by Former NIMH Director Thomas Insel: P4C : Time = Lives

By Thomas Insel on November 14, 2013

Last week I attended the Partners for Cure (P4C) meeting in New York. This is an annual meetup for an odd mix of academic scientists, advocacy groups, venture capitalists, social entrepreneurs, and biotech-pharma folks. In contrast to typical scientific meetings, which explore the “what” and the “how” of research, P4C is mostly about the “who” and the “when.” Specifically, who will deliver new cures and how do we speed up this process? The meeting was sponsored by a group called Faster Cures and driven by advocates, many fighting a fatal disease. Throughout the two-day meeting, they reminded scientists that “time = lives.”

But the theme that intrigued me was not the burning question of “when” but the disruptive question of “who.” For many of the P4C attendees, the traditional model of NIH funding to academic scientists followed by industry funding of translation to new treatments is not working. One participant described science that has not helped patients as “science fair projects.” Others disparaged a culture that focuses on papers rather than products. Noting that academic scientists are responsible for teaching, clinical care, and writing grants, many asked whether the academic engine for research is powered to go as fast and far as patients need. And for many the incentives of science in academia seemed disconnected from the growing unmet needs of patients. Success in academia is measured in number of papers, grant funding, and promotion to tenure. Success for patients means treatments and cures that offer a healthy, long life. As just one example, scientists have few incentives and some disincentives to share data or resources. For patients, progress may depend on the rapid sharing of information and crowd sourcing of tough problems.

What made the P4C meeting so inspiring was the chance to witness some of the new models that are changing our assumptions about “who” will deliver new cures. There were sessions on experiments in collaboration or crowd sourcing or new tools for a range of needs including cancer, Parkinson’s, Alzheimer’s, and serious mental illness. In fact, after four previous meetings, this was the first that included a session on serious mental illness. But aside from the disease focus, the message was that when nonprofits and companies and academia and government agencies work together, good things can happen.

Some of the most innovative experiments are being developed by disease advocacy groups:

In the Michael J. Fox Foundation’s Parkinson’s Data Challenge, researchers competed to develop the best way to benefit patients and clinicians by collecting objective, passive data on a large scale. (Passive data is generated as an incidental product of everyday activities, such as using a cell phone.) The winner, chosen from 630 entries by teams in 21 countries, was a mobile phone app that analyzed changes in voice quality to detect onset and progression of Parkinson’s disease.

The Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT), a joint project of academia, pharma, and nonprofits, amassed more than 8,600 de-identified subject records from 18 clinical trials on amyotrophic lateral sclerosis treatment into a single, harmonized dataset that is now being mined by scientists from more than 30 countries.

The Project Data Sphere Initiative is a big data initiative led by the Life Sciences Consortium, a task force of the CEO Roundtable on Cancer. The Initiative provides access to the data from the control arms of Phase III clinical trials. This dataset, based on records from thousands of patients, accelerates research by providing detailed information about the progression of various tumors.

A new company, Curious, is driving a patient-centric research movement by helping disease foundations develop platforms for data sharing and open access.

There were remarkable stories from a new generation of entrepreneurs. Halle Tecco, co-founder of Rock Health, talked about why she left Apple to begin a company investing in mobile apps for health. Jessica Richman, co-founder of uBiome, showed how the world’s largest crowd-funded citizen-science startup established a unique microbiome map for science. Josh Sommer, diagnosed with chordoma while an undergraduate at Duke, has built the Chordoma Foundation to unify and accelerate global research on this rare disease.

What became clear at P4C was that the “who” question is being answered in very creative ways. Using registries and sensors and crowd sourcing, patients are becoming drivers not just passengers on the research bus. The traditional model of government-funded research in academia followed by industry research to develop marketed treatments is being replaced by collaborations between nonprofits, academic scientists, and biotech with a goal of delivering treatments more quickly for more people. Science has become global, slowed only by the rate of data sharing and access. Because time = lives, we need to go faster. Thankfully, there is a new generation with some fresh ideas.